nesolicaftor

Nesolicaftor (PTI-428) is an investigational small molecule drug developed for the treatment of cystic fibrosis (CF). It acts as a CFTR amplifier, targeting the cystic fibrosis transmembrane conductance regulator (CFTR) protein, which is defective in people with CF. By increasing the amount of functional CFTR protein produced, nesolicaftor aims to improve chloride and water transport across cell membranes, thereby reducing thick mucus buildup and improving symptoms in patients. Unlike some other modulators that are effective only for specific mutations such as F508del, nesolicaftor has shown activity across a variety of different CF-causing mutations. It is being studied both as an add-on to existing approved therapies and as part of proprietary combination regimens[1][2][5][6].

Other names

nesolicaftor